Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 19 for:    Fatty Acid Oxidation Disorder
Previous Study | Return to List | Next Study

A Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03833128
Recruitment Status : Recruiting
First Posted : February 6, 2019
Last Update Posted : August 26, 2019
Sponsor:
Information provided by (Responsible Party):
Reneo Pharma Ltd

Tracking Information
First Submitted Date  ICMJE February 4, 2019
First Posted Date  ICMJE February 6, 2019
Last Update Posted Date August 26, 2019
Actual Study Start Date  ICMJE April 4, 2019
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 5, 2019)
Adverse Events [ Time Frame: Continous to Week 12 ]
Number of participants with Adverse Events (AEs) as a measure of safety and tolerability
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03833128 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders
Official Title  ICMJE An Open Label Study to Determine the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Subjects With Fatty Acid Oxidation Disorders (FAOD)
Brief Summary The purpose of this study is to assess REN001 safety in subjects with fatty acid oxidation disorders.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fatty Acid Oxidation Disorders
Intervention  ICMJE
  • Drug: Low Dose REN001
    Once daily for 12 weeks
  • Drug: High Dose REN001
    Once daily for 12 weeks
Study Arms  ICMJE
  • Experimental: Group 1
    REN001 Low Dose
    Intervention: Drug: Low Dose REN001
  • Experimental: Group 2
    REN001 High Dose
    Intervention: Drug: High Dose REN001
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 5, 2019)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Subjects must give written, signed and dated informed consent

Confirmed diagnosis of FAOD

A diagnostic acylcarnitine profile, in blood or cultured fibroblasts

A stable treatment regimen for at least 30 days prior to enrollment

Exclusion Criteria:

Unstable or poorly controlled disease

Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer

Have been hospitalized within 3 months prior to screening for any major medical event

Pregnant or nursing females

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Lynn Purkins, PhD +44 (0) 1304 809360 lpurkins@reneopharma.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03833128
Other Study ID Numbers  ICMJE REN001-102
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Reneo Pharma Ltd
Study Sponsor  ICMJE Reneo Pharma Ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Jerry Vockley, M.D., Ph.D. University of Pittsburgh Medical Center
PRS Account Reneo Pharma Ltd
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP